1. Field of the Invention
The present invention relates to the field of treating or preventing tissue deterioration, injury or damage due to a neuro-, muscular- or neuro-muscular-degenerative disease, or restoring tissue adversely affected by said disease.
2. Description of the Background Art
Neuro-degenerative diseases, muscular-degenerative diseases and neuro-muscular-degenerative diseases are debilitating diseases that can destroy memory, brain functions, muscle functions and other physiological functions of a subject. Such diseases may be genetic, or result from exposure by a subject to factors in the environment, contaminated food, and the like. Such diseases may include Alzheimer's disease, multiple sclerosis (MS), Lou Gehrig's disease (amytrophic lateral sclerosis or ALS), Parkinson's disease, spinal muscular atrophy (SMA), myasthenia gravis, autism, muscular dystrophy, transmissible spongiform encephalopathies (TSEs) including bovine spongiform encephalopathy (BSE), and the like.
Muscular dystrophies are genetic disorders characterized by progressive muscle wasting and weakness that begin with microscopic changes in the muscle. As muscles degenerate over time, the person's muscle strength declines.
Duchenne muscular dystrophy (DMD) is one of a group of muscular dystrophies characterized by the enlargement of muscles. DMD is one of the most prevalent types of muscular dystrophy and is characterized by rapid progression of muscle degeneration that occurs early in life. All are X-linked and affect mainly males—an estimated 1 in 3500 boys worldwide.
The gene for DMD, found on the X chromosome, encodes a large protein—dystrophin. Dystrophin is required inside muscle cells for structural support; it is thought to strengthen muscle cells by anchoring elements of the internal cytoskeleton to the surface membrane. Without it, the cell membrane becomes permeable, so that extracellular components enter the cell, increasing the internal pressure until the muscle cell “explodes” and dies. The subsequent immune response can add to the damage.
Becker muscular dystrophy (BMD) is a much milder version of DMD. Its onset is usually in the teens or early adulthood, and the course is slower and far less predictable than that of DMD.
In the U.S., there are over 84,000 patients with degenerative muscular dystrophy diseases.
There remains a need in the art for methods of treatment for treating, preventing, inhibiting or reducing tissue deterioration, injury or damage due to a neuro-, muscular- or neuro-muscular-degenerative disease, or for restoring tissue adversely affected by said disease.